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BMJ No 7114 Volume 315 Editorial Saturday 18 October 1997
High cost, low volume care: the case of haemophiliaReverting to central funding might be the only optionHaemophilia is a rare and expensive condition. In Britain it affects 5,418 males with factor VII deficiency and 1,109 with factor IX deficiency, and in 1994 they used 158 million units of factor VIII and 9 million of factor IX at an average cost of 30p per unit. Over the past 20 years the amount of clotting factor used per patient has increased, and both the quality of the clotting factors and methods of administration have improved. In theory the nature and level of treatment is specified in contracts between purchasers and providers, but at our centre, which cares for 14% of the haemophilic population of England and Wales, contract revenue is regularly outweighed by the cost of care. Our cost pressures are similar to those of any high cost, low volume clinical service in any general trust. Accumulating experience suggests that the present funding arrangements are failing; the danger is that such services will become a liability and be eliminated by both providers and purchasers. Clotting factor concentrate represents 50-80% of the total direct cost of haemophilia care.(1) Over the past 15 years the use of concentrate in Britain has risen threefold. For reasons of viral safety recombinant factor VIII is the treatment of choice(2); recombinant factor IX is also likely to become so once it is licensed. However, while intermediate purity plasma derived clotting factor costs 32p per unit and is exempt from value added tax, recombinant factor VIII costs 52p per unit and is liable to 17.5% VAT. Thus in our centre, where the median annual use of concentrate for an adult is 72,000 units, the annual cost per patient would be £23,000 for intermediate purity plasma derived concentrate but £44,000 (including VAT) for recombinant factor VIII. A further cost pressure has been changes in treatment strategy, particularly the introduction of prophylaxis for children. Traditionally a patient with severe haemophilia received clotting factor concentrate (30 IU/kg) after a bleed and could expect 30-35 bleeds per year.(3) Long term prophylactic regimens, introduced before any sign of joint damage, have benefited patients by preventing joint damage and improving the quality of life.(4) These regimens, however, require a fourfold increase in clotting factor use. Although in Britain the number of boys aged under 10 with severe haemophilia is small (only 385 in 1994), for our trust the cost of giving 31 of them prophylaxis with recombinant concentrate is about £2m. Furthermore, contracts for this care have to be negotiated with 16 health authorities. Perhaps the greatest difficulty, however, is the unpredictability of individual clotting factor requirements. For example, the concentrate required for a total knee replacement for haemophilic arthropathy could double the annual cost of treatment for a single patient. Although information on the cost effectiveness of prophylaxis is beginning to emerge,(5) there is no hard information on the benefits of using recombinant factors over plasma derived concentrate. Currently we rely on the biological plausibility that recombinant factors are likely to prove beneficial in the long term. Whether it is economically desirable to increase spending on patients now is open to argument, but within the constraints of an annual contracting round adopting a longer perspective is clearly difficult. Should we be investing in alternative ways of reducing costs, such as gene therapy and continuous infusion? What are the costs and benefits of liver transplantation, which can cure haemophilia?(6) Additional costs of iatrogenic infectionsIatrogenic problems add to the cost pressures. In 1979-86, 1,321 individuals with haemophilia in Britain were infected with HIV from clotting factor concentrate, and 560 are currently alive. Our centre looks after 70 of them. All concentrates are now sterilised and no new transmissions have occurred since 1986.(7) Such processes have added considerably to the cost of treatment, but there is good evidence that monoclonally purified products slow the deterioration of the immune system in HIV positive patients.(8) Although additional funding was provided to pay for placing patients on these high purity products, patients with end stage AIDS consume upwards of 50% more clotting factor than when they are asymptomatic.(9) It has been estimated that 25 years from seroconversion - that is, around the year 2008 for the haemophilic community - 20% will still remain AIDS free.(10) With the advent of triple antiviral therapy, the cost of drug treatment as well as a longer duration of life will add to the costs of caring for these patients. Most patients treated with large pool clotting factor concentrates between 1965 and 1985 were infected with hepatitis C virus. A fifth are coinfected with HIV, which accelerates the progression of the liver disease.(11) Many are treated with interferon, calculated at a lifetime cost of £70,555-£195,407.(12) In addition, since coagulation factors are synthesised in the liver, increased amounts of factors VII, VIII, and IX are required when the liver fails. These deaths are largely unpredictable but occur at a rate of two a year in our centre. There is no additional funding for concentrate in these circumstances. Contracting for this high cost service is made harder by the uneven geographical spread of patients. In 1994, 42 of 85 haemophilia centres treated fewer than 10 patients with severe disease; only three centres, including our own, treated more than 110. These three centres treated over half the 2368 patients needing clotting factor concentrates in 1994 in England and Wales. These escalating and unpredictable costs mean that expenditure on haemophilia treatment is often not covered within a block contract. As a result the trust and purchasers find it increasingly difficult to ensure that patients with haemophilia receive appropriate care while sustaining the level of service in other specialities. In the long term it may be necessary to revert to central funding for this rare, expensive, unpredictable, and lifelong condition and others like it. Christine Lee
Professor of haemophilia
Haemophilia Centre and Haemostasis Unit,
References
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